Ionis Pharmaceuticals has announced that the Food and Drug Administration has granted Orphan Drug designation to IONIS-HTT for the treatment of Huntington’s disease.

IONIS-HTT is the first target-directed therapy in clinical development designed to treat the underlying cause of Huntington’s disease, a rare, genetic neurological disorder.

The Gen. 2.0+ antisense drug directly targets the cause of Huntington’s disease by reducing the production of all forms of the huntingtin (HTT) protein. This mechanism offers a unique approach for the treatment of the disease.

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There is currently no effective disease modifying treatment, and current approaches only focus on managing some disease symptoms.


Ionis Pharmaceuticals press release

This article originally appeared on MPR